Award Data

PROJECT SUMMARY Congenital Myasthenic Syndromes (CMS) are a group of clinically similar neuromuscular transmission disorders that differ in their underlying genetic mutation. While some phenotypical variation exists, CMS are commonly characterized by muscle weakness (myasthenia) that worsens with physical exertion. Defects in Collagen Q (ColQ), a multidomain functional protein of the Neuro Muscula ...

For more than a decade, our work has focused on development of therapeutic interventions for viral- and bacterial-induced acute lung injury (ALI) and the more severe acute respiratory distress syndrome (ARDS). Significantly, we identified the Toll-like receptor 4 (TLR4) signaling pathway as key to the host response to influenza and secondary bacterial infection following influenza. We also identif ...

Project Summary There are 24,000 stillbirths per year in the United States, and 2.6 million stillbirths per year worldwide, with no significant decrease in the last decade. Abnormal fetal heart rhythms, such as long QT syndrome, are thought to play an important role in these deaths, and may cause 3- 10% of unexplained stillbirths. This is in addition to known fetal arrhythmias, which affect 1-3% o ...

Project Summary/Abstract for STTR Phase I Application: An immunotherapeutic IgY formulation against norovirus diarrhea Human norovirus (HuNoV) infects millions of people globally each year, causing diarrheal illness resulting in more than 200,000 deaths annually, particularly in the very young, the very old, and the immunocompromised. There is currently neither an effective vaccine nor effective t ...

ABSTRACTGlobally, more than 71 million people are infected with Hepatitis C virus (HCV), with 1-2 million new infections occurring each year. This major health concern necessitates the development of an effective vaccine. Since HCV rapidly accumulates mutations, vaccines must elicit the production of broadly neutralizing antibodies (bnAbs) in a reproducible fashion. The viral envelope E1E2 glycopr ...

PROJECT SUMMARY Microinjection technologies underlie many research and clinical applications that require gene and cell delivery, including studies and emerging treatments of neurological conditions (e.g., neurodegenerative diseases, traumatic brain injury, and cancer). Unfortunately, challenges associated with the microinjection tools by which the viral vectors and cells inherent in these applica ...

PROJECT SUMMARY We propose to develop a novel non-contact, label-free multimodality imager that provides real-time intraoperative identification of nerves within the surgical field-of-view to facilitate the prevention of unintended nerve damage (termed iatrogenic nerve injury) during surgical procedures, as they are a major source of postsurgical complications, e.g., chronic pain. In the United St ...

Abstract There is an unmet need to develop treatments for senile osteoporosis, a disorder characterized by an age-related reciprocal decrease in osteogenesis and increase in bone marrow fat. Senile osteoporosis resembles disuse osteopenia, suggesting its pathogenesis involves impaired bone mechanosensing. We discovered that the polycystin heterotrimeric complex (1PC1+3PC2) functions as a mechanose ...

Project SummaryEpilepsy is one of the most common brain disorders. Current drugs have limited efficacy. Identifying new targeted drugs that can be used as safer, more effective therapies is in urgent unmet need. Selective inhibition of the transient receptor potential canonical 3 (TRPC3) emerges as a novel strategy to impede epilepsy. However, the currently best selective TRPC3 inhibitor, Pyr3, ha ...

Project Summary Volume overload (congestion) occurs in patients with heart failure (HF) and is the leading cause of hospitalization in the elderly worldwide. Unfortunately, clinical signs and symptoms; laboratory values; and existing diagnostic tools are unreliable in providing accurate assessment of volume status. Using a unique physiologic signal, Non-Invasive Venous waveform Analysis (NIVA), Vo ...